In short
Autologous Peripheral Blood is an investigational cell therapy being studied in a Phase 1 trial for children, adolescents, and young adults with high-grade central nervous system (CNS) tumours. The trial, authorised in the EU Clinical Trials Information System, will enrol 30 participants and is testing whether modified immune cells given directly into the central nervous system are safe.
Key points
- Phase 1 trial in children, adolescents, and young adults with high-grade CNS tumours
- 30 participants planned for enrolment
- Trial status: Authorised in the EU Clinical Trials Information System
- Tests CART-NKG2D cells and NKIL15 cells given loco-regionally into the CNS
- Primary focus: safety, including cytokine release syndrome and neurotoxicity
- Administered as a solution for injection directly into the central nervous system
What is autologous peripheral blood?
Autologous Peripheral Blood is the short label for autologous peripheral blood-derived CD45RA- T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against NKG2-D type II integral membrane protein. In the trial record, this investigational product is referred to as CART45RA-NKG2D cells. It is a type of cell therapy made from a person's own immune cells, which are collected from the blood, modified in a laboratory, and then returned to the same person. The substance is not an authorised medicine; it is being investigated in a clinical trial.
How does autologous peripheral blood work?
The modified immune cells are designed to carry a chimeric antigen receptor (CAR) that recognises a protein called NKG2-D. This receptor is intended to help the modified T-cells find and attack tumour cells. The trial also includes a second product, NKIL15, which consists of unexpanded autologous peripheral blood adult differentiated NK cells stimulated with IL-15. Both products are being tested for their ability to act against aggressive brain tumours when delivered directly into the central nervous system.
Which conditions is it studied for?
- Central nervous system tumours — the trial focuses on high-grade CNS tumours in children, adolescents, and young adults.
How is autologous peripheral blood given?
Both investigational products are prepared as a solution for injection and are administered loco-regionally, meaning they are delivered directly into the central nervous system rather than into the bloodstream.
What do the clinical trials show?
One Phase 1 trial is registered in the EU Clinical Trials Information System with the identifier 2026-525876-24-00. The trial is currently authorised and plans to enrol 30 participants.
The primary objective is to determine the safety of the two products when given loco-regionally to children and young adults with high-grade CNS tumours. The trial measures the incidence and severity of adverse reactions, serious adverse events, and grade 3 or higher toxicities at day 48. It also records the incidence and grade of cytokine release syndrome (CRS) and neurotoxicity, including whether participants need intensive care or treatment with tocilizumab or corticosteroids.
Because this is a Phase 1 trial, the main question is whether the treatment is safe, not yet whether it is effective. The trial is interventional, meaning participants receive the investigational products as part of the study.